CompletedPhase 1Phase 2NMOSDNCT00787722

What it found

Results posted, no main test reported

Results are posted in the registry, but without a statistical test of the main measure. See the numbers below.

What was tested

Treatment tested

  • Hematopoietic Stem Cell Transplantation
  • Cyclophosphamide anhydrous
  • Filgrastim

    Granulocyte colony stimulating factor receptor agonist

  • rATG
  • 2-mercaptoethanesulfonic acid
  • Rituximab

    B-lymphocyte antigen CD20 binding agent

  • Methylprednisolone

    Glucocorticoid receptor agonist

Results by group

Survival

6 months, 1 year, 2 year, 3 year, 4 year, 5 year - after the transplant

GroupValueIntervalNumber analysed
Hematopoietic Stem Cell Transplantation13 Participants13
Hematopoietic Stem Cell Transplantation12 Participants13
Hematopoietic Stem Cell Transplantation12 Participants13
Hematopoietic Stem Cell Transplantation11 Participants13
Hematopoietic Stem Cell Transplantation11 Participants13
Hematopoietic Stem Cell Transplantation11 Participants13

Values as posted on ClinicalTrials.gov. Outcomes measured differently across trials are not directly comparable.

Safety

GroupParticipants at riskSerious adverse eventsDeaths
Hematopoietic Stem Cell Transplantation131 (7.7%)1

As posted on ClinicalTrials.gov. Serious adverse events include any serious health event during the trial, whether or not the treatment caused it.

What it measured

Main measures

  • Survival

    6 months, 1 year, 2 year, 3 year, 4 year, 5 year - after the transplant

Other measures (5)

  • Quality of Life (QOL) Short Form - 36 (SF-36)

    pre-transplant 12mo and 5 years

    Quality of life (reported by participants)
  • Post HSCT Immune -Modulating Medication and Relapse

    Pre transplant and 6 months, 1 year, 2 year, 3 year, 4 year and 5 year after transplant

    Relapses
  • Number of Patients Who Require No Device Assistance for Ambulation

    6 months, 1 year, 2 year, 3 year, 4 year, 5 year - after the transplant

  • Disability Score: Expanded Disability Status Scale (EDSS)

    pretransplant 6 month, 5 year

    Disability progression
  • NMO-IgG Aquaporin- 4 Autoantibody Titer

    Pretransplant and 5 year Post Transplant

    Immune response

Who could take part

NMOSD · 16 to 65

Inclusion Criteria: * Age between 16-65, at the time of pretransplant evaluation * An established diagnosis of Devic's disease (more than one acute attack) * NMO- IgG aquaporin-4 autoantibody positive Exclusion Criteria: * Paraplegia or quadriplegia and legal blindness (defined as visual acuity of 20/200 or less in the better eye with the best correction possible) * Any illness that in the opinion of the investigators would jeopardize the ability of the patient to tolerate aggressive chemotherapy * Prior history of malignancy except localized basal cell, squamous skin cancer or carcinoma in situ of the cervix. Other malignancies for which the patient is judged to be cured, such as head and neck cancer, or breast cancer will be considered on an individual basis * Positive pregnancy test * Inability or unwillingness to pursue effective means of birth control. Effective birth control is defined as 1) refraining from all acts of vaginal intercourse (ABSTINENCE); 2) consistent use of birth control pills; 3) injectable birth control methods (Depo-provera, Norplant); 4) tubal sterilization or male partner who has undergone vasectomy; 5) placement of an intrauterine device (IUD); or 6) use, with every act of intercourse, of diaphragm with contraceptive jelly and/or condoms with contraceptive foam * Failure to willingly accept or comprehend irreversible sterility as a side effect of therapy * forced expiratory volume at one (FEV1) / forced vital capacity (FVC) \< 60% of predicted after bronchodilator therapy (if necessary) * Diffusing capacity of lung for carbon monoxide (DLCO) \< 50% of predicted * Resting left ventricular ejection fraction (LVEF) \< 50 % * Serum creatinine > 2.0 mg/dl * Known hypersensitivity to mouse, rabbit, or E. Coli derived proteins * Presence of metallic objects implanted in the body that would preclude the ability of the patient to safely have MRI exams * Bilirubin > 2.0 mg/dl * Platelet count \< 100,000/ul or absolute neutrophil count (ANC) \< 1000/ul * Psychiatric illness, mental deficiency or cognitive dysfunction making compliance with treatment or informed consent impossible * Active infection except asymptomatic bacteriuria * Inability to give informed consent * HIV positive * Transaminases > 3x of normal limits, liver cirrhosis

As registered. Criteria can change, so ask the trial team or your neurologist.

About this trial

In the sponsor's words

This study is designed to examine whether treating Devic's disease patients with high dose cyclophosphamide together with rabbit antithymocyte globulin (rATG)/rituximab (drugs which reduce the function of the immune system), followed by return of previously collected patient's stem cells will result in improvement in Devic's disease. Stem cells are undeveloped cells that have the capacity to grow into mature blood cells, which normally circulate in the blood stream. The purpose of the intense chemotherapy is to destroy the cells in patient's immune system, which may be causing his/her disease. The purpose of the stem cell infusion is to produce a normal immune system that will no longer attack patient's body. The purpose of study is to examine the safety and efficacy of this treatment. The drugs used in this study treatment are drugs for commonly used for immune suppression.

Papers

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Related trials

History

    1. Started

      Oct 2009

    2. Main results due

      Nov 2018

    3. Finished

      Nov 2018

    4. Results posted

      Feb 2020

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